Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (October 2024)
30 slides from Moleculin Biotech's investor presentation, Oct 2, 2024
Slide 1: October 2024 Investor Presentation
Slide 2: Disclaimer
Disclaimer All statements contained herein other than statement of historical fat, including statements regarding our future resis of operations and financial positon fur business strategy and plans, and our objectives for fture operations, are forward-looking statements, The words “believe,” “may!”
Slide 3: Moleculin key facts and management experience
7 FDA Approvals 2. Big Pharma Exits Moleculin 14 Clinical Trials $ 5 Million in Recent +9 Management Investment Years of Drug 200 Development Experience
Slide 4: Technology Portfolio
Technology Portfolio Program Indication ese ‘Annamycin R/R AML otal Initiating Next-Gen Anthracyclie srs tung Mets we1066 ‘Adult GBM STAT Inhibitor Pediatric Brain Tumors ites GBM; Virology Anti-metabolite
Slide 5: Anthracycline use across cancers
ob /., = $F MOLECULIN ¥ Breast cancers = 32% AML patients = 50% Lymphomas = 70% Childhood cancers = 60%
Slide 6: Annamycin Attributes
Annamycin Attributes ‘No vesicant activity Developed in (safer to handle and collaboration with A administer) ‘and licensed from, Composition ‘MD Anderson. ) of Matter . icy Avoids Cross Ditters Non- 20n0 Resistance incidence (10%) of alopecia vs Dox (60- Cardiotoxic : wath currently px te anthracyc
Slide 7: Approved Therapies are Successful for Only ~40% of AML Patients
Approved Therapies are Successful for Only ~40% of AML Patients* Venetoclax Regimen Ven-Aza ——Gene-Targeted FitPatients UnfitElderly 55% -65% Sub-population —""(50%) (60%) Eligible Durable CR% (MUNNSE% 37% =21% AIl AML benefit? 14% 15% 1% Cardiotox; de Stillleaves. Limited to novolacquired significa
Slide 8: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) 22 4 10 1 14 1 Subjects Evaluabie Not Sonate 2 9 = CCl tle 67.5 (19-78) 56.5 (19-69) 71 (53 - 78) 4 69.5 (53-78) fission 1 Conca Rentals 8 (36%) 2 (60%) 5 (50%) 1 BGS) mission 1 Congien heres 9 (41%) 2(60%) 6 (60%) ] 7 (60%) Partial Response (PR) 1 2 Re Rel
Slide 9: Clinical History Supporting Activity in Leukemia
Clinical History Supporting Activity in Leukemia Subjects at RP2D 8 5 22 RR AML/ALL ALL AML AML Fotal of 35 Prior Lines of theray Race inca) 1-5 (2) 2-8 (4) 0-5 (1) subjects treated at over 10 unique sites Regimen Single Agent ‘Single Agent Combination with 15 (43%) achieving # of Sites 4 (Us) 5 (EU
Slide 10: MiRAcLE
MiRAC LESS Moleculin R/R AML AnnAraC Clinical Evaluation Phase 3 pivotal clinical trial designed to seek accelerated new drug approval for Annamycin in the treatment of relapsed or refractory acute myeloid leukemia. Targeted to begin recruiting in the first quarter of 2025
Slide 11: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation Annamy: Unblinded 190mg/m* + Data Readout Annamycin Optimum Establish Jose + HDAC 230 mel + HIDAC *Criteria: (n=30) Efficacy Safety Tolerability Control Arm HIDAC + placebo (n=30) Primary Endpoint = CR rate at “1 month n sub
Slide 12: The Bar for Approval is Low
The Bar for Approval is Low Complete Remission (CR) HIDAC CR rates Annamycin ire well iecineeted NDA to be based on CR f rate in 2"4 138% line subjects at ~1 month osc? wos 2 ‘fonaac? Amman MirosTrial Clase Trial AlKComers ands Sed ne o% 1—Miros Trial, 81% 2° line patients, Konopleva et al, Blood Ad
Slide 13: Estimated Regulatory Timeline
Estimated Regulatory Timeline 2025 | 2026 | 2027 2028 2029 Enrollment Blinded CR Breakthrough Enroliment Accelerated Approval Begins : Designation (on Primary Endpoint >. ae 2024 Enrollment Begins arms (190 mg, 230 mg, control; 4 7 Interim review to select optimum of trial (Optimum Dose vs. control;
Slide 14: Expected Visibility at Part A Midpoint (2H25)
Expected Visibility at Part A Midpoint (2H25) Historical Expected Blinded CR Response Midpoint Rate at Part A Rates Responders Midpoint Consistent with Historical Rates
Slide 15: AnnAraC Should Increase 2nd Line AML CRs 2-Fold or More
AnnAraC Should Increase 2nd Line AML CRs 2-Fold or More 60.0% i 1 / so.0% | 50.0% ~13% of 2L AML Patients Expected to ! 1 Achieve CR 1 i 40.0% 1 C . if i 30.0% ' i 1 1 20.0% 1 i 1 ' 10.0% | 58% | 23% 1.7% 2.4% | 13%} 1 1 0.0% a = mv -. ve | 1 Gilteritinib Enasidenib —IvosidenibOlutasidenib Gemtuzuma
Slide 16: Potential Asset Value
Potential Asset Value Approved P Ziftomenit 199/174 cR 38% 19%/25% 35% 18% 24% CRe 48% 23%/33% 40% 25% 47% population 50% 50% 15-23% 3087 2a 30% Revenue? $128M $28 ~$150M $1.5B $28 ~$1.5B “$1.98 Valuation Exit! N/A Exit Market Market N/A ‘een ) sete oe ele
Slide 17: Soft Tissue Sarcoma Mets to the Lungs
Soft Tissue Sarcoma Mets to the Lungs “We don’t expect to see these kinds of responses in STS patients with lung metastases who have stopped responding to 1* line therapy...let alone in 4" line!” STS KOL * Preliminary results, subject to final clinical study report which is expected to be issued in
Slide 18: The Full Annamycin Opportunity
The Full Annamycin Opportunity Potential for Significant Upside as Pancreatic Liver 13,500 PFS and OS rivaling neous STS Lung Metastases PRS ey es om STS subjects? Endometrial Lung : 20,000 104K Initial Annamycin Pte nylod Colorectal Target Indications} Leukemia fete rman ther TOTAL INCIDENCE 101K T
Slide 19: Cash to Fund Operations into the First Quarter of 2025
Cash to Fund Operations into the First Quarter of 20251 Nasdaq: MBRX ~$15M Cash Balance? ~$14.4M Market Cap? ~5.6M Shares Fully Diluted Outstanding* ~58K — Month of August Avg. Daily Trading Volume®
Slide 20: Estimated Regulatory Timeline
Estimated Regulatory Timeline 2025 | 2026 | 2027 2028 2029 Enrollment Blinded CR Breakthrough Enroliment Accelerated Approval Begins Designation (on Primary Endpoint 2024 Enrollment Begins arms (190 mg, 230 mg, control; 4 7 Interim review to select optimum of trial (Optimum Dose vs. control; for the
Slide 21: MiRAcLE
MiRAC LESS Moleculin R/R AML AnnAraC Clinical Evaluation Phase 3 pivotal clinical trial designed to seek accelerated new drug approval for Annamycin in the treatment of relapsed or refractory acute myeloid leukemia. Targeted to begin recruiting in the first quarter of 2025
Slide 22: Appendix
Slide 23: Performance of AML Therapies in Line
Performance of AML Therapies in 24 Line CLAVELA: International Randomized Phase Ill Study of Elacytarabine Versus Investigator Choice in Patients with Relapsed/Refractory Acute Myeloid Leukemia 381 Elacytarabine Therapies compared: R/R AML (compared with) + high-dose © low-dose cytarabine cytarabine
Slide 24: AML Clinical History
AML Clinical History Phase 1: MB-104, Phase 1/2: MB-105 Phase 1/2: MB-106, N fc MBIVATION THERAPY Ne7 N=20 N= 22alllines (0-6), N= 10 (2° ine) 117% CRi (at suboptimal dosing) ‘Median tines of prior therapy ‘All subjects (N-22) 41% CR (TT) Dosing imited by FDA Lifetime Median age of 240 me/m?(RPD2) 2
Slide 25: Durability, MRD, Prior Therapies
Durability, MRD, Prior Therapies ~7 months median durability and climbing 78% MRD negative urabilty of Remission p= Continuing f= Prior Lines of Therapy © subject relapsed, likely ue cessation of treatment due to infection, Continues with F/U Subject died from pneumonia following infection 1) M105 d
Slide 26: MB-106 Response by Genotype and Mutation
MB-106 Response by Genotype and Mutation TF’s or PR CRe’s bea 31 cHRM cons 89% (nz8) of CRe responses [= — 7, — ana came from subjects with “generally poor prognosis cytogenetics and mutations” RUNX1/1T1 F381 Annamycin appears to be “genotype/mutation agnostic” Note tiple mutations or abnormalities,
Slide 27: Annamycin Demonstrates Efficacy in STS Lung Metastases
Annamycin Demonstrates Efficacy in STS Lung Metastases (MB-107) - As Reported in Feb 2024 Demonstrated Stable Disease After Demonstrated Improvement with Dose < 330 mg/m? ‘Two Treatment Cycles and Fewer Prior Therapies All Subjects Preliary MO- ry as of Preliminary and Confidential
Slide 28: Science Advisors
Science Advisors Waldemar Priebe, PhD RMwAIEE MD Anderson Cancer Center Mayo Clink Founding Scientist & SAB Chair Hematology Oncology Dr. Martin Tallman Dr. Jorge Cortes Northwestern University ‘Augusta University Dr. Michael Andreef Dr. Giovanni Martinelli MD Anderson Cancer Center Bologna Universi
Slide 29: Global Network
Global Network All technology licensed from MD Anderson Cancer Center (MDAC) Supports continuing preclinical research on our technology at MDAC close to $1M per year ‘Active contractors in US, EU and Asia for drug production and distribution as well as for clinical trial management Past & current ex
Slide 30: Upcoming Milestones
Upcoming Milestones \ Annamycin AML Annamycin STS Lung Mets Begin contracting with MIRACLE trial sites First subject treated in MIRACLE trial Recruitment Update (n=45) Data Readout (n=90) unblinded and Optimum Dose set for MIRACLE trial Begin enrollment of 3" ine subjects in MIRACLE2 Enrollment ends
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